MedTrace Logo

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

NCT04323098 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 22

Last updated 2025-10-08

Study results available
· View outcomes & findings →

Summary

This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in combination with prophylactic corticosteroids in patients with severe hemophilia A.

Conditions

Interventions

BIOLOGICAL

valoctocogene roxaparvovec

Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A

Sponsors & Collaborators

Principal Investigators

  • Medical Monitor, MD · BioMarin Pharmaceutical

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-12-08
Primary Completion
2023-01-27
Completion
2025-05-08
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Brazil
  • Taiwan

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04323098 on ClinicalTrials.gov